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Companies involved in gene therapy of hematological disorders 139
In utero gene therapy 139
Fetal gene transfer techniques 139
Animal models of fetal gene therapy 140
Potential applications of fetal gene therapy 140
Fetal gene therapy for cystic fibrosis 141
Fetal intestinal gene therapy 141
Hearing disorders 141
Potential of gene therapy 142
Vectors for gene therapy of hearing disorders 142
Auditory hair cell replacement and hearing improvement by gene therapy 143
Kidney diseases 143
End-stage renal disease 143
Methods of gene delivery to the kidney 144
Gene transfer into kidney by adenoviral vectors 144
Non-viral gene transfer to the kidneys 144
Gene transfer into the glomerulus by HVJ-liposome 145
Bone marrow stem cells for renal disease 145
Mesangial cell therapy 145
Liposome-mediated gene transfer into the tubules 146
Gene transfer to tubules with cationic polymer polyethylenimine 146
Gene therapy in animal experimental models of renal disease 146
Genetic manipulations of the embryonic kidney 147
Antisense intervention in glomerulonephritis 147
Gene therapy for renal fibrosis 147
Use of genetically engineered cells for uremia due to renal failure 148
Concluding remarks 148
Liver disorders 148
Techniques of gene delivery to liver 149
Direct injection of DNA into liver 149
Local gene delivery by isolated organ perfusion 150
Liposome-mediated direct gene transfer 150
Retroviral vector for gene transfer to liver 150
Adenoviral vectors for gene transfer to liver 150
Receptor-mediated approach 151
Cell therapy for liver disorders 151
Transplantation of genetically modified hepatocytes 151
Genetically modified hematopoietic stem cells 152
Gene therapy by ex vivo transduce
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